Children's Hospital of Wisconsin, Milwaukee, Wisconsin, USA.
J Heart Lung Transplant. 2009 Dec;28(12):1335-40. doi: 10.1016/j.healun.2009.06.028. Epub 2009 Sep 26.
Restrictive cardiomyopathy (RCM) in children often has a progressive nature, with a high risk of clinical deterioration and death. Heart transplantation (HTx) is a widely accepted therapy that offers long-term survival, but criteria for and outcomes after listing have not been well defined.
A multi-institutional, prospective, event-driven data registry of 3,147 patients aged < 18 years listed for HTx from January 1993 to December 2006 was used to assess risk factors and survival of 145 listed RCM patients.
Mean age at listing was 8.1 years, with 44% listed as United Network of Organ Sharing status 1, 33% on inotropic support, 10% on a ventilator, and 5% on mechanical support. At 1 year, 82% of these patients survived to HTx, whereas 9% died waiting. Univariate risk factors for death while waiting included younger age (p < 0.001), ventilator dependence (p < 0.001), status 1 (p < 0.001), and inotrope usage (p < 0.001). Use of multiple support devices at listing (ventilator, extracorporeal membrane oxygenation, ventricular assist device, intraaortic balloon pump) was also an important risk factor for early phase death while waiting (relative risk; 9.01, p < 0.0001). Survival after listing was 63% at 10 years and compared favorably with survival for non-cardiomyopathy patients (p = 0.01).
Children with RCM awaiting HTx have a generally low waitlist mortality and reasonable overall survival. Children requiring mechanical support and infants had a significantly higher risk of death while waiting. Further study is warranted to identify factors important in determining the optimal timing of listing in children with RCM before the need for inotropic or mechanical support.
儿童限制性心肌病（RCM）通常具有进行性，临床恶化和死亡风险高。心脏移植（HTx）是一种广泛接受的治疗方法，可提供长期生存，但尚未明确列出的标准和结果。
使用 1993 年 1 月至 2006 年 12 月期间接受 HTx 治疗的 3147 名年龄<18 岁的患者的多机构，前瞻性，事件驱动的数据登记处，评估了 145 名列出的 RCM 患者的危险因素和生存率。
列出的平均年龄为 8.1 岁，有 44％列为 UNOS 状态 1，33％接受正性肌力支持，10％接受呼吸机支持，5％接受机械支持。在 1 年内，82％的患者存活至 HTx，而 9％的患者在等待中死亡。等待期间死亡的单因素危险因素包括年龄较小（p<0.001），呼吸机依赖（p<0.001），状态 1（p<0.001）和正性肌力支持（p<0.001）。列出时使用多种支持设备（呼吸机，体外膜氧合，心室辅助设备，主动脉内球囊泵）也是等待期间早期死亡的重要危险因素（相对风险；9.01，p<0.0001）。列出后的 10 年生存率为 63％，与非心肌病患者的生存率相比具有优势（p=0.01）。
等待 HTx 的 RCM 患儿的等待名单死亡率普遍较低，整体生存率合理。需要机械支持的儿童和婴儿等待期间死亡的风险明显更高。需要进一步研究，以确定在需要正性肌力或机械支持之前，RCM 患儿列出的最佳时机的重要因素。
